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Atlas / Drug / Cytarabine

Cytarabine

drug
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Also known as AlexanAlexan 100Ara-cAra-cytidineArabinocytosineArabinosyl cytosineAracytidineAracytinAracytineCitarabinaCytarabine component of vyxeosCytarabine liposomeCytarabinosideCytarabinumCytosarCytosar-uDepocytDepocyteNSC-287459

Summary

Cytarabine (CHEMBL803) is an approved small-molecule antineoplastic agent (ATC L01BC01); indicated across 80 conditions including acute lymphoblastic leukemia and acute myeloid leukemia; with CIViC clinical evidence for 17 variant-indication associations (e.g. IKZF1 Deletion in b-lymphoblastic leukemia/lymphoma).

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: L01BC01
  • Indications: 80 conditions
  • Clinical trials: 1,089
  • Precision-oncology evidence (CIViC): 17 variant–indication associations
  • Chemistry: 243.22 Da · C9H13N3O5

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL803
NameCytarabine
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID6253
ChEBICHEBI:28680
ATCL01BC01
Molecular formulaC9H13N3O5
Molecular weight243.22
InChIKeyUHDGCWIWMRVCDJ-CCXZUQQUSA-N

SMILES: C1=CN(C(=O)N=C1N)[C@H]2[C@H]([C@@H]([C@H](O2)CO)O)O

IUPAC name: 4-amino-1-[(2R,3S,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]pyrimidin-2-one

ChEBI definition: A pyrimidine nucleoside in which cytosine is attached to D-arabinofuranose via a β-N1-glycosidic bond. Used mainly in the treatment of leukaemia, especially acute non-lymphoblastic leukaemia, cytarabine is an antimetabolite antineoplastic agent that inhibits the synthesis of DNA. It also has antiviral and immunosuppressant properties.

Pharmacological roles (ChEBI): antineoplastic agent, antiviral agent, immunosuppressive agent.

Other ChEBI roles (chemical / environmental): antimetabolite.

Also known as: Alexan, Alexan 100, Ara-c, Ara-cytidine, Arabinocytosine, Arabinosyl cytosine, Aracytidine, Aracytin, Aracytine, Citarabina, Cytarabine, Cytarabine component of vyxeos

Parent form; salt/anhydrous children: CHEMBL1256472

Patent coverage: 51,109 distinct patent families (202,889 SureChEMBL compound mentions), from 3 matched compound structure(s). One matched structure accounts for 202,867 (100%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 9 (assay-derived). Sample: Prelamin-A/C, 4’-phosphopantetheinyl transferase ffp, Nuclear receptor coactivator 3, Nuclear receptor coactivator 1, Latent membrane protein 1, Thyroid hormone receptor beta, Serine/threonine-protein kinase mTOR, Tegument protein VP16, E3 ubiquitin-protein ligase Mdm2.

Bioactivity

ChEMBL activities: 8 potent at pChembl ≥ 5 of 12 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
MDM27.7IC5020nMCHEMBL_ACT_18773015
LMNA7.4Potency39.8nMCHEMBL_ACT_3647269
MTOR7.13Potency73.6nMCHEMBL_ACT_4787615
P032306.13AC50736nMCHEMBL_ACT_7471811
MTOR6.03Potency926.8nMCHEMBL_ACT_3919490
P032305.96AC501100nMCHEMBL_ACT_7362694
MTOR5.88Potency1309nMCHEMBL_ACT_4522866
NCOA35.12IC507533nMCHEMBL_ACT_8260712

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

80 indications (11 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

IndicationTrial phaseMONDOEFO
acute lymphoblastic leukemia4MONDO:0004967EFO:0000220
acute myeloid leukemia4MONDO:0018874EFO:0000222
neoplasm4MONDO:0005070EFO:0000616
acute myeloblastic leukemia without maturation4MONDO:0005224EFO:0003027
lymphoid leukemia4MONDO:0005402EFO:0004289
acute myeloblastic leukemia with maturation4MONDO:0020320EFO:0003028
tumor of meninges4MONDO:0016743EFO:0003851
lymphoma3MONDO:0005062EFO:0000574
leukemia3MONDO:0005059EFO:0000565
acute promyelocytic leukemia3MONDO:0012883EFO:0000224
mantle cell lymphoma3MONDO:0018876EFO:1001469
B-cell acute lymphoblastic leukemia3MONDO:0004947EFO:0000094
neoplasm of mature B-cells3MONDO:0004949EFO:0000096
Hodgkins lymphoma3MONDO:0004952EFO:0000183
myelodysplastic syndrome3MONDO:0018881EFO:0000198
T-cell acute lymphoblastic leukemia3MONDO:0004963EFO:0000209
acute monocytic leukemia3MONDO:0007896EFO:0000221
acute myelomonocytic leukemia M43MONDO:0018871EFO:0000223
chronic myeloid leukemia3MONDO:0011996EFO:0000339
diffuse large B-cell lymphoma3MONDO:0018905EFO:0000403
melanoma3MONDO:0005105EFO:0000756
malaria3MONDO:0005136EFO:0001068
plasma cell myeloma3MONDO:0009693EFO:0001378
acute megakaryoblastic leukemia3MONDO:0018872EFO:0003025
acute basophilic leukemia3MONDO:0019458EFO:0003029
myelodysplastic syndrome with single lineage dysplasia3MONDO:0005272EFO:0003802
non-Hodgkin lymphoma3MONDO:0018908EFO:0005952
granulocytic sarcoma3MONDO:0006237EFO:1000286
Langerhans cell histiocytosis3MONDO:0018310EFO:1000318
myeloid sarcoma3MONDO:0006861EFO:1001052
acute erythroid leukemia3MONDO:0017858EFO:1001257
chronic myelomonocytic leukemia3MONDO:0020311EFO:1001779
myeloproliferative neoplasm3MONDO:0020076EFO:0002428
myelodysplastic syndrome with excess blasts3MONDO:0019454EFO:0003811
breast neoplasm3MONDO:0021100MONDO:0007254
follicular lymphoma3MONDO:0018906MONDO:0018906
acute biphenotypic leukemia3MONDO:0020322MONDO:0019460
graft versus host disease3MONDO:0013730MONDO:0013730
thrombotic disease3MONDO:0000831MONDO:0000831
myeloid leukemia3MONDO:0004643MONDO:0004643
sarcoma2MONDO:0005089EFO:0000691
B-cell chronic lymphocytic leukemia2MONDO:0004948EFO:0000095
peripheral T-cell lymphoma, not otherwise specified2MONDO:0004964EFO:0000211
Burkitt lymphoma2MONDO:0007243EFO:0000309
medulloblastoma2MONDO:0007959EFO:0002939
anaplastic large cell lymphoma2MONDO:0020325EFO:0003032
relapsing-remitting multiple sclerosis2MONDO:0005314EFO:0003929
juvenile myelomonocytic leukemia2MONDO:0011908EFO:1000309
stiff-person syndrome2MONDO:0008491EFO:0007498
neuromyelitis optica2MONDO:0019100EFO:0004256
myasthenia gravis2MONDO:0009688EFO:0004991
Guillain-Barre syndrome, familial2MONDO:0007691EFO:0009538
blast phase chronic myelogenous leukemia, BCR-ABL1 positive2MONDO:0006115EFO:1000131
central nervous system neoplasm2MONDO:0006130EFO:1000158
aseptic meningitis2MONDO:0006662EFO:1000823
opsoclonus-myoclonus syndrome2MONDO:0015247EFO:1001383
neutropenia2MONDO:0001475MONDO:0001475
ovarian cancer2MONDO:0008170MONDO:0008170
hematopoietic and lymphoid system neoplasm2MONDO:0002334MONDO:0044881
systemic lupus erythematosus2MONDO:0007915MONDO:0007915
plasma cell neoplasm2MONDO:0004959EFO:0000200
CD4+/CD56+ hematodermic neoplasm2MONDO:0019467EFO:0010580
plasma cell leukemia1MONDO:0018689EFO:0006475
glioblastoma1MONDO:0018177EFO:0000519
essential thrombocythemia1MONDO:0005029EFO:0000479
acquired polycythemia vera1MONDO:0009891EFO:0002429
primary myelofibrosis1MONDO:0009692MONDO:0044903
brain neoplasm1MONDO:0021211EFO:0003833
paraganglioma1MONDO:0000448EFO:1000453

11 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 1,089.

Phase distribution

PhaseTrials
PHASE2457
PHASE3195
PHASE1189
PHASE1/PHASE2135
Not specified49
PHASE434
PHASE2/PHASE325
EARLY_PHASE15

Top trials by phase / activity

NCTPhaseStatusTitle
NCT05518383PHASE4RECRUITINGB-cell Mature Non-Hodgkin’s Lymphoma Treatment Protocol in Children and Adolescents 2021
NCT06289673PHASE4RECRUITINGIdentification of Necessary Information for Treatment Induction in Newly Diagnosed Acute Lymphoblastic Leukemia/Lymphoma
NCT06763666PHASE4NOT_YET_RECRUITINGCLAG+VEN vs CLAG in the Treatment of Relapsed/Refractory AML
NCT00180115PHASE4COMPLETEDAML96 - Risk-Adapted and Randomized Postremission-Therapy for Adult Acute Myeloid Leukemia Patients
NCT00180128PHASE4UNKNOWNAIDA2000 - Risk-Adapted Therapy for Patients With Acute Promyelocytic Leukemia
NCT00198978PHASE4COMPLETEDGerman Multicenter Trial for Treatment of Elderly Patients With Newly Diagnosed Acute Lymphoblastic Leukemia
NCT00198991PHASE4COMPLETEDGerman Multicenter Trial for Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Adults (07/2003)
NCT00199004PHASE4COMPLETEDTrial for Treatment of Adult Patients With Standard Risk Acute Lymphoblastic Leukemia With Chemotherapy and Rituximab
NCT00199017PHASE4COMPLETEDGerman Multicenter Trial for the Treatment of Newly Diagnosed T-lymphoblastic Lymphoma in Adults
NCT00199056PHASE4COMPLETEDGerman Multicenter Trial for Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Adults (06/99)
NCT00199069PHASE4COMPLETEDGerman Multicenter Trial for Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Adults (05/93)
NCT00199082PHASE4COMPLETEDNewly Diagnosed Mature B-ALL, Burkitt’s Lymphoma and Other High-grade Lymphoma in Adults
NCT00199095PHASE4COMPLETEDTreatment of Elderly Patients (>65 Years) With Acute Lymphoblastic Leukemia
NCT00199147PHASE4UNKNOWNEfficacy of G-CSF-Priming in Elderly AML Patients
NCT00408278PHASE4COMPLETEDTreatment of Newly Diagnosed Patients With Acute Promyelocytic Leukemia (PETHEMA LPA 2005)
NCT00464217PHASE4COMPLETEDTreatment of the Acute Myeloblastic Leukaemia in Patients Over 65 Years
NCT00487448PHASE4COMPLETEDSMD_FLAG-IDA_98: FLAG-IDA in Induction Treatment of High Risk Myelodysplastic Syndromes or Secondary Acute Myeloblastic Leukemia
NCT00488709PHASE4COMPLETEDFludarabine, Cytarabine, Topotecan in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia
NCT00494897PHASE4COMPLETEDPETHEMA LAL-RI/96: Treatment for Patients With Standard Risk Acute Lymphoblastic Leukemia
NCT00526175PHASE4COMPLETEDLAL-BR/2001: Study Treatment to Low Risk ALL
NCT00526305PHASE4COMPLETEDLAL-Ph-2000: Treatment of Acute Lymphoblastic Leukemia Chromosome Philadelphia Positive
NCT00526409PHASE4COMPLETEDLAL-AR-N-2005:Study Treatment for Children High Risk Acute Lymphoblastic Leukemia
NCT00853008PHASE4COMPLETEDTreatment of High Risk Adult Acute Lymphoblastic Leukemia
NCT01191541PHASE4COMPLETEDCytarabine (Ara-C) in Children With Acute Promyelocytic Leukemia (APL)
NCT01358201PHASE4UNKNOWNPETHEMA LAL-07FRAIL: All Treatment In Fragile Patients Ph’ Negative Over 55 Years
NCT01358253PHASE4COMPLETEDRituximab Plus Chemotherapy for CD20+ Adult Acute Lymphoblastic Leukemia
NCT01366898PHASE4UNKNOWNProtocol For the Treatment Acute Lymphoblastic Leukemia With Ph ‘Negative in Elderly Patients (> 55 Years)
NCT02024308PHASE4UNKNOWNAML1-ETO Acute Myeloid Leukemia With Fludarabine and Cytarabine Chemotherapy
NCT02036489PHASE4COMPLETEDPethema LAL-RI/2008: Treatment for Patients With Standard Risk Acute Lymphoblastic Leukemia
NCT02200978PHASE4COMPLETEDA Study for Improving the Outcome of Childhood Acute Promyeloid Leukemia
NCT02858804PHASE4COMPLETEDEDOCH Alternating With DHAP for New Diagnosed Younger MCL
NCT02894645PHASE4UNKNOWNMalaysia-Singapore Acute Lymphoblastic Leukemia 2010 Study
NCT02926586PHASE4COMPLETEDFludarabine and Cytarabine Versus High-dose Cytarabine for CBF-AML
NCT03026842PHASE4UNKNOWNDecitabine Versus Conventional Chemotherapy for Maintenance Therapy of Acute Myeloid Leukemia With t(8;21)
NCT02003222PHASE3ACTIVE_NOT_RECRUITINGCombination Chemotherapy With or Without Blinatumomab in Treating Patients With Newly Diagnosed BCR-ABL-Negative B Lineage Acute Lymphoblastic Leukemia
NCT02101853PHASE3ACTIVE_NOT_RECRUITINGBlinatumomab in Treating Younger Patients With Relapsed B-cell Acute Lymphoblastic Leukemia
NCT02112916PHASE3ACTIVE_NOT_RECRUITINGCombination Chemotherapy With or Without Bortezomib in Treating Younger Patients With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia or Stage II-IV T-Cell Lymphoblastic Lymphoma
NCT02205762PHASE2/PHASE3ACTIVE_NOT_RECRUITINGLCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With Langerhans Cell Histiocytosis
NCT02339740PHASE3ACTIVE_NOT_RECRUITINGTretinoin and Arsenic Trioxide in Treating Patients With Untreated Acute Promyelocytic Leukemia
NCT02405676PHASE2/PHASE3ACTIVE_NOT_RECRUITINGBNHL-2015 for Children or Adolescents in China

Clinical evidence (CIViC)

Variant × indication × effect (17 predictive associations from 17 curated evidence items):

VariantIndicationEffectTherapyLevelCIViC
IKZF1 DeletionB-lymphoblastic Leukemia/lymphomaSensitivity/ResponseMethotrexate + Daunorubicin + Cytarabine + Fludarabine + ImatinibCIViC BEID7366
RUNX1 MutationAcute Myeloid LeukemiaResistanceCytarabineCIViC BEID411
WT1 Exon 7 MutationAcute Myeloid LeukemiaResistanceDaunorubicin + CytarabineCIViC BEID139
FLT3 D835VAcute Myeloid LeukemiaSensitivity/ResponseSunitinib + CytarabineCIViC DEID3009
PRPS1 L191FB-lymphoblastic Leukemia/lymphomaSensitivity/ResponseCytarabineCIViC DEID7911
PRPS1 N144SB-lymphoblastic Leukemia/lymphomaSensitivity/ResponseCytarabineCIViC DEID7910
NT5C2 R238WChildhood Acute Lymphocytic LeukemiaResistanceCytarabine + Gemcitabine + Doxorubicin + PrednisoloneCIViC DEID7816
NT5C2 R367QChildhood Acute Lymphocytic LeukemiaResistanceCytarabine + Gemcitabine + Prednisolone + DoxorubicinCIViC DEID7815
NT5C2 S445FChildhood Acute Lymphocytic LeukemiaResistanceCytarabine + Doxorubicin + Gemcitabine + PrednisoloneCIViC DEID7817
PRPS1 A190TB-lymphoblastic Leukemia/lymphomaResistanceCytarabine + Asparaginase + Methotrexate + DaunorubicinCIViC DEID7928
PRPS1 D183EB-lymphoblastic Leukemia/lymphomaResistanceAsparaginase + Methotrexate + Cytarabine + DaunorubicinCIViC DEID7927
PRPS1 K176NB-lymphoblastic Leukemia/lymphomaResistanceDaunorubicin + Cytarabine + Methotrexate + AsparaginaseCIViC DEID7926
PRPS1 S103NB-lymphoblastic Leukemia/lymphomaResistanceMethotrexate + Asparaginase + Daunorubicin + CytarabineCIViC DEID7925
PRPS1 S103TB-lymphoblastic Leukemia/lymphomaResistanceMethotrexate + Asparaginase + Daunorubicin + CytarabineCIViC DEID7924
PRPS1 T303SB-lymphoblastic Leukemia/lymphomaResistanceAsparaginase + Daunorubicin + Cytarabine + MethotrexateCIViC DEID7929
ZEB1 ExpressionMantle Cell LymphomaResistanceDoxorubicin + Cytarabine + GemcitabineCIViC DEID899
NPM1 W288FSAcute Myeloid LeukemiaSensitivity/ResponseDaunorubicin + Cytarabine + EtoposideCIViC EEID153

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 33 clinical and 130 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.2
BioBTree
v2.0.2

Sources 17

This page pulls from 17 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclinical_trialsefogtopdb_interactiongtopdb_ligandhgncmeshmondopatent_compoundpharmgkbpharmgkb_guidelinepubchemuniprot